Page last updated: 2024-11-07

prednisone and Muscular Dystrophies, Limb-Girdle

prednisone has been researched along with Muscular Dystrophies, Limb-Girdle in 6 studies

Prednisone: A synthetic anti-inflammatory glucocorticoid derived from CORTISONE. It is biologically inert and converted to PREDNISOLONE in the liver.
prednisone : A synthetic glucocorticoid drug that is particularly effective as an immunosuppressant, and affects virtually all of the immune system. Prednisone is a prodrug that is converted by the liver into prednisolone (a beta-hydroxy group instead of the oxo group at position 11), which is the active drug and also a steroid.

Muscular Dystrophies, Limb-Girdle: A heterogenous group of inherited muscular dystrophy that can be autosomal dominant or autosomal recessive. There are many forms (called LGMDs) involving genes encoding muscle membrane proteins such as the sarcoglycan (SARCOGLYCANS) complex that interacts with DYSTROPHIN. The disease is characterized by progressing wasting and weakness of the proximal muscles of arms and legs around the HIPS and SHOULDERS (the pelvic and shoulder girdles).

Research Excerpts

ExcerptRelevanceReference
"In this single center, open label pilot study, once-weekly prednisone was safe and well tolerated."3.11An Open Label Exploratory Clinical Trial Evaluating Safety and Tolerability of Once-Weekly Prednisone in Becker and Limb-Girdle Muscular Dystrophy. ( Ajroud-Driss, S; Casey, P; Joslin, BC; McNally, EM; Zelikovich, AS, 2022)
" Herein, we assessed the efficacy of steroid dosing on sarcolemmal repair, muscle function, histopathology, and the regenerative capacity of primary muscle cells."1.46Intermittent Glucocorticoid Dosing Improves Muscle Repair and Function in Mice with Limb-Girdle Muscular Dystrophy. ( Demonbreun, AR; McNally, EM; Page, PG; Quattrocelli, M; Salamone, IM; Warner, JL, 2017)
"Alendronate treatment did not ameliorate muscle degeneration, but demonstrated a limited enhancement on muscle function test."1.43Glucocorticoid Steroid and Alendronate Treatment Alleviates Dystrophic Phenotype with Enhanced Functional Glycosylation of α-Dystroglycan in Mouse Model of Limb-Girdle Muscular Dystrophy with FKRPP448L Mutation. ( Blaeser, A; Bollinger, LE; Cox, MD; Harper, AD; Lu, P; Lu, QL; Luckie, TM; Madden, KL; Richardson, SM; Shah, SN; Sparks, S; Sun, Y; Wu, B, 2016)
"Prednisone therapy was associated with: (i) functional improvement of overall motor disability, in upper limbs of DMD (P < 0."1.33The effects of glucocorticoid therapy on the inflammatory and dendritic cells in muscular dystrophies. ( Abu-Dief, EE; Hamed, SA; Hussein, MR; Kamel, NF; Kandil, MR; Mostafa, MG, 2006)

Research

Studies (6)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's2 (33.33)29.6817
2010's3 (50.00)24.3611
2020's1 (16.67)2.80

Authors

AuthorsStudies
Zelikovich, AS1
Joslin, BC1
Casey, P1
McNally, EM2
Ajroud-Driss, S1
Quattrocelli, M1
Salamone, IM1
Page, PG1
Warner, JL1
Demonbreun, AR1
Dadgar, S1
Wang, Z1
Johnston, H1
Kesari, A1
Nagaraju, K1
Chen, YW1
Hill, DA1
Partridge, TA1
Giri, M1
Freishtat, RJ1
Nazarian, J1
Xuan, J1
Wang, Y1
Hoffman, EP1
Wu, B1
Shah, SN1
Lu, P1
Richardson, SM1
Bollinger, LE1
Blaeser, A1
Madden, KL1
Sun, Y1
Luckie, TM1
Cox, MD1
Sparks, S1
Harper, AD1
Lu, QL1
Godfrey, C1
Escolar, D1
Brockington, M1
Clement, EM1
Mein, R1
Jimenez-Mallebrera, C1
Torelli, S1
Feng, L1
Brown, SC1
Sewry, CA1
Rutherford, M1
Shapira, Y1
Abbs, S1
Muntoni, F1
Hussein, MR1
Hamed, SA1
Mostafa, MG1
Abu-Dief, EE1
Kamel, NF1
Kandil, MR1

Clinical Trials (4)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy[NCT04054375]Phase 220 participants (Actual)Interventional2019-07-01Completed
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)[NCT03439670]Phase 2121 participants (Actual)Interventional2018-06-29Completed
A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)[NCT05185622]Phase 254 participants (Anticipated)Interventional2022-03-21Recruiting
A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy[NCT05166109]Phase 239 participants (Anticipated)Interventional2022-07-07Recruiting
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Muscle Imaging

MRI of leg muscles to measure changes in muscle fat percentage. The data point was collected by taking fat percentage at 6 months minus fat percentage at baseline with the following equation: (([final fat percentage - initial fat percentage]/initial fat percentage) * 100%)). All participants were included, both ambulatory and nonambulatory, with all genetic subtypes included. Five participants didn't have an MRI scan at 6 months and therefore were not included. Muscles imaged were analyzed for muscle fat changes from baseline to 6 months. Data is limited in interpretation due to various muscle groups in both ambulatory and non-ambulatory patients. (NCT04054375)
Timeframe: Baseline, 6 months

Interventionpercent of change from baseline (Mean)
Weekly Steroid-14

10 Meter Run Timed

time in seconds to walk/run 10 meters , less time to run indicates greater motor function (NCT04054375)
Timeframe: Baseline, Month 6

Interventionseconds (Mean)
Baseline6 months
Steroid Treatment7.326.67

6 Minute Walk Test

number of meters walked in 6 minute period. Higher values indicate more motor function. (NCT04054375)
Timeframe: Baseline, Month 6

Interventionmeters (Mean)
Baseline6 months
Steroid Group386410

Bone Density

"whole dexa body scan to assess bone density with Z scores (more negative z score indicates increased risk for fractures).~Z-score of 0 represents the population mean, and is the average bone density. Positive scores indicate greater bone density and negative scores indicate decreased bone density, which could be clinically correlated with osteoporosis." (NCT04054375)
Timeframe: Baseline, 6 months

Interventionz-score (Mean)
baseline6 months
Weekly Steroid-1.64-1.65

Brooke Scale Score

upper extremity assessment, scoring between 1- 6, lower score indicates more upper extremity function (NCT04054375)
Timeframe: Baseline, Month 6

Interventionscores on a scale (Mean)
baseline6 months
Weekly Steroid33

Creatine Kinase

units/L, 0-unlimited, higher scores indicate worse outcome (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

InterventionU/L (Mean)
BaselineEnd
Weekly Steroid15741047

Fasting Glucose

mg/dL, 0-unlimited, higher score indicates worse outcome (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

Interventionmg/dL (Mean)
BaselineEnd
Weekly Steroid93102

Fasting Lipid Profile

cholesterol levels - mg/dL, higher levels indicate worse outcomes (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

Interventionmg/dL (Mean)
BaselineEnd
Weekly Steroid182185

Functional Assessments - NSAD Change

"Northstar Assessment for Dysferlinopathy~- score out of 58, range from 0 to 58, higher score indicates greater functional ability." (NCT04054375)
Timeframe: Baseline, Month 6

Interventionscore on a scale (Mean)
baseline6 months
Weekly Steroid18.418.6

Functional Assessments - Upper Limb Strength

"Grip strength of the total force (Newtons) in both hands.~Participants attempted 3 trials in the right hand that was then averaged to create a right-hand average force score.~Then, the participants attempted 3 trials in the left hand that was then averaged to create a left-hand average force score.~The right-hand average force score was added to the left-hand average force score to create a total grip strength score." (NCT04054375)
Timeframe: Baseline and 6 months

InterventionForce (Newtons) (Mean)
Baseline6 months
Weekly Steroid3941

HbgA1c

% , 0-100, higher score indicates worse outcome (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

Intervention% A1c (Mean)
BaselineEnd
Weekly Steroid5.25.3

Lean Mass %

whole body dexa scans to assess lean mass % (0- 100 %). Increase lean mass % is the desired outcome. (NCT04054375)
Timeframe: Baseline, 6 months

Interventionpercentage (Mean)
baseline6 months
Weekly Steroid37.538.1

Muscle Strength Test

Manual motor testing of the right knee flexion muscle group. (NCT04054375)
Timeframe: baseline, 6 months

InterventionUnits on scale (Mean)
Baseline6 months
Weekly Steroid33

Respiratory Changes

Force Vital Capacity (% of predicted value), decrease in FVC indicates declining respiratory function. (NCT04054375)
Timeframe: Baseline, 6 months

Intervention% Expected (Mean)
BaselineEnd
Weekly Steroid8079

Vignos Scale Score

Lower extremity assessment, score from 1-10, lower score indicates more function. (NCT04054375)
Timeframe: Baseline, Month 6

Interventionscores on a scale (Mean)
baseline6 months
Steroid Treatment Group55

Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline

Vamorolone at 6.0mg/kg/day vs. placebo group in change from baseline to the Week 24 assessment (NCT03439670)
Timeframe: 24 weeks

InterventionRises/Seconds (Mean)
Treatment Group 1-.007
Treatment Group 20.054

Trials

1 trial available for prednisone and Muscular Dystrophies, Limb-Girdle

ArticleYear
An Open Label Exploratory Clinical Trial Evaluating Safety and Tolerability of Once-Weekly Prednisone in Becker and Limb-Girdle Muscular Dystrophy.
    Journal of neuromuscular diseases, 2022, Volume: 9, Issue:2

    Topics: Drug Administration Schedule; Humans; Muscular Dystrophies, Limb-Girdle; Muscular Dystrophy, Duchenn

2022

Other Studies

5 other studies available for prednisone and Muscular Dystrophies, Limb-Girdle

ArticleYear
Intermittent Glucocorticoid Dosing Improves Muscle Repair and Function in Mice with Limb-Girdle Muscular Dystrophy.
    The American journal of pathology, 2017, Volume: 187, Issue:11

    Topics: Animals; Dystrophin; Glucocorticoids; Membrane Proteins; Mice; Muscle, Skeletal; Muscular Dystrophie

2017
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
    The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1

    Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma

2014
Glucocorticoid Steroid and Alendronate Treatment Alleviates Dystrophic Phenotype with Enhanced Functional Glycosylation of α-Dystroglycan in Mouse Model of Limb-Girdle Muscular Dystrophy with FKRPP448L Mutation.
    The American journal of pathology, 2016, Volume: 186, Issue:6

    Topics: Adrenal Cortex Hormones; Alendronate; Animals; Blotting, Western; Bone Density; Bone Density Conserv

2016
Fukutin gene mutations in steroid-responsive limb girdle muscular dystrophy.
    Annals of neurology, 2006, Volume: 60, Issue:5

    Topics: Anti-Inflammatory Agents; DNA Mutational Analysis; Dystroglycans; Exons; Female; Glycosylation; Huma

2006
The effects of glucocorticoid therapy on the inflammatory and dendritic cells in muscular dystrophies.
    International journal of experimental pathology, 2006, Volume: 87, Issue:6

    Topics: Adolescent; Adult; Analysis of Variance; Biopsy; Child; Child, Preschool; Dendritic Cells; Female; G

2006